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Clinical Trials in the USA / NCT06246513
Active, not recruiting Phase 3

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513 · tracked via the Priya Life Science USA tracker
Sponsor
Sarepta Therapeutics, Inc.
Phase
Phase 3
Started
2024-01-15
Last updated
2026-03-02

Condition(s) studied

Limb-girdle Muscular Dystrophy

Investigational drug(s) / intervention(s)

SRP-9003Glucocorticoid

SRP-9003: Solution for single IV infusion

Glucocorticoid: Oral tablet (prophylactic)

Study summary

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).

Eligibility

Sex
ALL
Min age
4 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Cohort 1, only ambulatory participants: * Able to walk without assistive aid * 10MWR \<30 seconds * NSAD ≥25 * Cohort 2, only non-ambulatory participants: * 10MWR ≥30 seconds or unable to perform * PUL 2.0 entry scale score ≥3 * Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations * Able to cooperate with muscle testing * Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers \<1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay. Exclusion Criteria: * Left ventricular ejection fraction \< 40% or clinical signs and/or symptoms of cardiomyopathy * Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation * Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment * Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4) Other inclusion/exclusion criteria apply.

Primary outcome measure(s)

Trial sites (10)

FacilityCityRegionStatus
University of California, San Diego-Altman Clinical and Translational Research Institute La Jolla California
Nationwide Childrens Hospital Columbus Ohio
The Children's Hospital of Philadelphia Philadelphia Pennsylvania
Children's Hospital of The King's Daughter Norfolk Virginia
University Hospital Leuven (UZ Leuven) Leuven Vlaams Brabant
NMRC Gent (UZ Gent) Ghent Belgium
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum Essen North Rhine-Westphalia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Sant Joan de Deu Barcelona Spain
Newcastle University Newcastle upon Tyne United Kingdom

More Sarepta Therapeutics, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06246513 on ClinicalTrials.gov ↗ ← All trials in the USA